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GeneE
10 sources retrieved · Most recent: April 2026 · Index updated 15 days ago
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L2HGDH
L-2-hydroxyglutarate dehydrogenase
Chromosome 14 · 14q21.3
NCBI Gene: 79944Ensembl: ENSG00000087299.13HGNC: HGNC:20499UniProt: Q9H9P8
68PubMed Papers
21Diseases
0Drugs
45Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
small molecule metabolic process(S)-2-hydroxyglutarate dehydrogenase activitymitochondrionmembraneL-2-hydroxyglutaric acidurianeurodegenerative diseasegenetic disorderlysosomal storage disease
✦AI Summary

L2HGDH is a mitochondrial inner membrane enzyme that catalyzes the oxidation of L-2-hydroxyglutarate (L-2-HG) to α-ketoglutarate (αKG) 1. The enzyme contains FAD and NAD(P)-binding domains essential for its catalytic activity 2. L2HGDH deficiency causes L-2-hydroxyglutaric aciduria (L2HGA), a rare autosomal recessive neurometabolic disorder characterized by systemic L-2-HG accumulation 3. Affected individuals present with progressive neurological symptoms including psychomotor retardation, cerebellar ataxia, seizures, macrocephaly, and abnormal white matter changes on brain MRI 34. Mechanistically, L-2-HG accumulation impairs αKG-dependent histone and DNA demethylases (KDM5, TET enzymes), disrupting epigenetic regulation 5. This leads to excessive histone methylation, MYC oncogene activation, neural progenitor cell hyperproliferation, and defective neuronal differentiation 5. Mouse models demonstrate that L2HGDH loss causes leukoencephalopathy, neuroinflammation, and late-onset neurodegeneration 6. Clinically, diagnosis relies on elevated urinary/plasma L-2-HG levels, MRI findings, and L2HGDH mutational analysis 3. Over 70 pathogenic mutations have been identified, including frameshift and nonsense mutations causing protein truncation 74. L2HGA represents a metabolic disease where epigenetic dysregulation, rather than simple metabolite toxicity, drives neurological pathology.

Sources cited
1
L2HGA is autosomal recessive with neurological manifestations; diagnosis via MRI, biochemistry, and L2HGDH mutational analysis
PMID: 20052767
2
Frameshift mutations in L2HGDH truncate FAD/NAD(P)-binding domain; L-2-HG accumulates in urine
PMID: 29980873
3
L-2-HG impairs neuronal differentiation by inhibiting KDM5 demethylases and activating MYC through histone methylation changes
PMID: 40667135
4
L2HGDH is mitochondrial membrane enzyme catalyzing L-2-HG oxidation to αKG; contains FAD-binding and substrate-binding domains
PMID: 37995940
5
L2HGA caused by L2HGDH mutations; L-2-HG accumulates in plasma and cerebrospinal fluid
PMID: 19911013
6
L2HGA presents with cerebellar ataxia, psychomotor retardation, seizures, macrocephaly; identified by nonsense mutations and deletions
PMID: 37378753
7
L2hgdh knockout mice show white matter abnormalities, gliosis, impaired neurogenesis, and late-onset neurodegeneration
PMID: 28137912
Disease Associationsⓘ21
L-2-hydroxyglutaric aciduriaOpen Targets
0.83Strong
neurodegenerative diseaseOpen Targets
0.51Moderate
genetic disorderOpen Targets
0.45Moderate
lysosomal storage diseaseOpen Targets
0.37Weak
mitochondrial diseaseOpen Targets
0.37Weak
goutOpen Targets
0.35Weak
hypertensionOpen Targets
0.33Weak
type 2 diabetes mellitusOpen Targets
0.31Weak
kidney failureOpen Targets
0.30Weak
peripheral arterial diseaseOpen Targets
0.29Weak
cataractOpen Targets
0.27Weak
Abnormality of metabolism/homeostasisOpen Targets
0.27Weak
Varicose veinsOpen Targets
0.22Weak
atherosclerosisOpen Targets
0.12Weak
nonpapillary renal cell carcinomaOpen Targets
0.10Weak
peripheral vascular diseaseOpen Targets
0.08Suggestive
neoplasmOpen Targets
0.06Suggestive
behavioral variant of frontotemporal dementiaOpen Targets
0.05Suggestive
amyotrophic lateral sclerosisOpen Targets
0.05Suggestive
renal cell carcinomaOpen Targets
0.05Suggestive
L-2-hydroxyglutaric aciduriaUniProt
Pathogenic Variants45
NM_024884.3(L2HGDH):c.1115del (p.Met372fs)Pathogenic
L-2-hydroxyglutaric aciduria
★★☆☆2026→ Residue 372
NM_024884.3(L2HGDH):c.368A>G (p.Tyr123Cys)Likely pathogenic
L-2-hydroxyglutaric aciduria
★★☆☆2026→ Residue 123
NM_024884.3(L2HGDH):c.208C>T (p.Arg70Ter)Pathogenic
L-2-hydroxyglutaric aciduria|not provided
★★☆☆2025→ Residue 70
NM_024884.3(L2HGDH):c.528G>T (p.Glu176Asp)Pathogenic
not provided|L-2-hydroxyglutaric aciduria
★★☆☆2025→ Residue 176
NM_024884.3(L2HGDH):c.530_533delinsATT (p.Pro177fs)Pathogenic
not provided|L-2-hydroxyglutaric aciduria
★★☆☆2025→ Residue 177
NM_024884.3(L2HGDH):c.1A>G (p.Met1Val)Pathogenic
L-2-hydroxyglutaric aciduria|Inborn genetic diseases
★★☆☆2024→ Residue 1
NM_024884.3(L2HGDH):c.751C>T (p.Arg251Ter)Pathogenic
L-2-hydroxyglutaric aciduria
★★☆☆2024→ Residue 251
NM_024884.3(L2HGDH):c.1064+1G>ALikely pathogenic
L-2-hydroxyglutaric aciduria
★★☆☆2024
NM_024884.3(L2HGDH):c.293A>G (p.His98Arg)Pathogenic
L-2-hydroxyglutaric aciduria|not provided
★★☆☆2024→ Residue 98
NM_024884.3(L2HGDH):c.959del (p.Asp320fs)Pathogenic
L-2-hydroxyglutaric aciduria
★★☆☆2024→ Residue 320
NM_024884.3(L2HGDH):c.905C>T (p.Pro302Leu)Pathogenic
L-2-hydroxyglutaric aciduria|not provided
★★☆☆2024→ Residue 302
NM_024884.3(L2HGDH):c.1003C>T (p.Arg335Ter)Pathogenic
L-2-hydroxyglutaric aciduria
★★☆☆2023→ Residue 335
NM_024884.3(L2HGDH):c.256+1G>APathogenic
not provided|L-2-hydroxyglutaric aciduria
★★☆☆2022
NM_024884.3(L2HGDH):c.829C>T (p.Arg277Ter)Pathogenic
L-2-hydroxyglutaric aciduria|not provided
★★☆☆2022→ Residue 277
NM_024884.3(L2HGDH):c.903T>G (p.Tyr301Ter)Pathogenic
L-2-hydroxyglutaric aciduria
★★☆☆2020→ Residue 301
NM_024884.3(L2HGDH):c.844C>T (p.Arg282Trp)Likely pathogenic
L-2-hydroxyglutaric aciduria
★☆☆☆2025→ Residue 282
NM_024884.3(L2HGDH):c.699dup (p.Asp234fs)Pathogenic
L-2-hydroxyglutaric aciduria
★☆☆☆2025→ Residue 234
NM_024884.3(L2HGDH):c.467G>T (p.Gly156Val)Likely pathogenic
L-2-hydroxyglutaric aciduria
★☆☆☆2024→ Residue 156
NM_024884.3(L2HGDH):c.802G>T (p.Glu268Ter)Pathogenic
L-2-hydroxyglutaric aciduria
★☆☆☆2024→ Residue 268
NM_024884.3(L2HGDH):c.944del (p.Phe315fs)Pathogenic
L-2-hydroxyglutaric aciduria
★☆☆☆2024→ Residue 315
View on ClinVar ↗
Related Genes
ADHFE1Shared pathway100%GPT2Shared pathway100%CSProtein interaction93%FHProtein interaction93%GLUD1Protein interaction89%ALDH4A1Protein interaction86%
Tissue Expression6 tissues
Brain
100%
Heart
97%
Liver
91%
Ovary
42%
Bone Marrow
30%
Lung
27%
Gene Interaction Network
Click a node to explore
L2HGDHADHFE1GPT2CSFHGLUD1ALDH4A1
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted · UniProt Q9H9P8
View on AlphaFold ↗
Constraintⓘ
LOEUFⓘ
0.87LoF Tolerant
pLIⓘ
0.00Tolerant
Observed/Expected LoF0.64 [0.47–0.87]
RankingsWhere L2HGDH stands among ~20K protein-coding genes
  • #6,910of 20,598
    Most Researched68
  • #1,426of 5,498
    Most Pathogenic Variants45
  • #7,696of 17,882
    Most Constrained (LOEUF)0.87
Genes detectedL2HGDH
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
An overview of L-2-hydroxyglutarate dehydrogenase gene (L2HGDH) variants: a genotype-phenotype study.
PMID: 20052767
Hum Mutat · 2010
1.00
2
A novel compound heterozygous mutation of the L2HGDH gene in a Chinese boy with L-2-hydroxyglutaric aciduria: case report and literature review.
PMID: 29980873
Neurol Sci · 2018
0.90
3
L-2-hydroxyglutarate impairs neuronal differentiation through epigenetic activation of
PMID: 40667135
bioRxiv · 2025
0.80
4
MYC Regulation of D2HGDH and L2HGDH Influences the Epigenome and Epitranscriptome.
PMID: 32101699
Cell Chem Biol · 2020
0.70
5
Identification of novel L2HGDH gene mutations and update of the pathological spectrum.
PMID: 19911013
J Hum Genet · 2010
0.60